How does Vyondys 53 work?
Vyondys 53 (golodirsen) is a medication used to treat Duchenne muscular dystrophy (DMD) in patients who have a mutation of the DMD gene that is amenable to exon 53 skipping.
The DMD or dystrophin gene encodes an essential protein called dystrophin, which is needed for muscle integrity. People with DMD usually have a deletion mutation in exons 43-55. This means that their version of the dystrophin gene is missing some of the genetic material that they need to make a functional dystrophin protein.
Muscles lacking functional dystrophin protein get damaged when they contract. Without properly functioning dystrophin protein, normal muscle tissue is replaced by fibrous tissue and fat.
Vyondys 53 is an antisense oligonucleotide administered by intravenous infusion that works by binding to exon 53 of dystrophin pre-mRNA, which results in this exon being skipped over during mRNA processing. Skipping of exon 53 allows cells to join different exons together to produce a dystrophin protein that is shorter but has some function.
References
- LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012-. Golodirsen. [Updated 2020 Feb 27]. Available from: https://www.ncbi.nlm.nih.gov/books/NBK555332/?report=reader#_NBK555332_pubdet_
- Action Duchenne. Exon Skipping. [Accessed November 8, 20201]. Available from: https://www.actionduchenne.org/therapy-approaches/exon-skipping/.
- Food and Drug Administration (FDA). Vyondys 53. [Accessed November 8, 2021]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/211970s002lbl.pdf.
Read next
What are the new drugs for Duchenne muscular dystrophy?
The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45, Viltepso, Vyondys 53, Exondys 51, Elevidys, Emflaza and Duvyzat. They include classes such as antisense oligonucleotides, a glucocorticoid, a gene therapy and a histone deacetylase (HDAC) inhibitor. Continue reading
How long does it take for Vyondys 53 to work?
Results from a small phase I/II clinical trial show that treatment with Vyondys 53 increases dystrophin protein levels within 48 weeks of starting treatment. Continue reading
How is Vyondys 53 administered?
Vyondys 53 (golodirsen) is administered by intravenous (IV) infusion, which means it’s administered into a vein through a needle.
Vyondys 53 is given by a healthcare professional once a week. It is administered over 35 to 60 minutes via an in-line 0.2 micron filter. An indwelling venous access catheter is generally required. Continue reading
Related medical questions
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- What types of DMD can Exondys 51 be used for?
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Drug information
- Vyondys 53 Information for Consumers
- Vyondys 53 prescribing info & package insert (for Health Professionals)
- Side Effects of Vyondys 53 (detailed)
Related support groups
- Vyondys 53 (4 questions, 3 members)
- Duchenne Muscular Dystrophy (11 questions, 4 members)